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Clinical trial condition

Genetic Diseases, Inborn

Explore clinical trials studying Genetic Diseases, Inborn. Study availability and eligibility vary by location and protocol.

12,530 public trials 2,140 recruiting studies
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Genetic Diseases, Inborn trial results

Recruiting

Neurologic Stem Cell Treatment Study

NCT02795052

Conditions
ALS, Alzheimer Disease, Amyotrophic Lateral Sclerosis, +94 more
Locations
  • MD Stem Cells Westport, Connecticut, United States
  • MD Stem Cells Coral Springs, Florida, United States
  • The Saudi-German Hospital Dubai, United Arab Emirates
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Recruiting

A Phase 1 Open-Label Study of the Safety of Intravenous Allogeneic Neonatal Mesenchymal Cells (nMSCs) in Young Adult (1A) and Pediatric (1B) Patients With Dilated Cardiomyopathy (DCM)

NCT06464588

Conditions
Cardiomegaly, Cardiomyopathies, Cardiomyopathy, Dilated, +6 more
Locations
  • Grady Memorial Hospital Atlanta, Georgia, United States
  • Hughes Spalding Children's Hospital Atlanta, Georgia, United States
  • Emory University Hospital Atlanta, Georgia, United States
  • Arthur M. Blank Hospital Atlanta, Georgia, United States
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Recruiting

Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

NCT06716814

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +16 more
Locations
  • Sobi Investigational Site Plovdiv, Bulgaria
  • Sobi Investigational Site Sofia, Bulgaria
  • Sobi Investigational Site Brest, France
  • Sobi Investigational Site Bron, France
View all 21 locations
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Recruiting

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

NCT06904261

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Emory Genetics Atlanta, Georgia, United States
  • University of Minnesota Masonic Children's Hospital Minneapolis, Minnesota, United States
  • Atrium Health Levine Children's Hospital Charlotte, North Carolina, United States
  • Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States
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Recruiting

A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B

NCT07285460

Conditions
Blood Coagulation Disorders, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, +7 more
Locations
  • The Luskin Orthopaedic Institute for Children- Site Number : 8400013 Los Angeles, California, United States
  • The Center for Inherited Blood Disorders- Site Number : 8400009 Orange, California, United States
  • Cure 4 The Kids Foundation- Site Number : 8400001 Las Vegas, Nevada, United States
  • Hackensack Meridian Health - Hackensack University Medical Center- Site Number : 8400008 Hackensack, New Jersey, United States
View all 30 locations
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